Improving single injection CSF delivery of AAV9-mediated gene therapy for SMA: a dose-response study in mice and nonhuman primates (Q35153080)

25358252

6 August 2017

title

Improving single injection CSF delivery of AAV9-mediated gene therapy for SMA: a dose-response study in mice and nonhuman primates (English)

1 reference

6 August 2017

0 references

2

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7 June 2020

Alessandra Govoni

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7 June 2020

Arthur Burghes

13

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7 June 2020

author name string

Kathrin Meyer

1

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7 June 2020

Leah Schmelzer

3

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7 June 2020

Lyndsey Braun

4

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7 June 2020

Vicki McGovern

5

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7 June 2020

Shibi Likhite

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7 June 2020

Olivia Michels

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Julie Fitzgerald

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Pablo Morales

10

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7 June 2020

Kevin D Foust

11

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Jerry R Mendell

12

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7 June 2020

Brian K Kaspar

14

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7 June 2020

language of work or name

1 reference

31 October 2014

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6 August 2017

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7 June 2020

volume

23

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7 June 2020

issue

3

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7 June 2020

page(s)

477-487

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Intrathecal gene therapy corrects CNS pathology in a feline model of mucopolysaccharidosis I

7 June 2020

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Strong cortical and spinal cord transduction after AAV7 and AAV9 delivery into the cerebrospinal fluid of nonhuman primates

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Global CNS gene delivery and evasion of anti-AAV-neutralizing antibodies by intrathecal AAV administration in non-human primates

28 July 2018

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Intramuscular scAAV9-SMN injection mediates widespread gene delivery to the spinal cord and decreases disease severity in SMA mice.

28 July 2018

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28 July 2018

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A single administration of morpholino antisense oligomer rescues spinal muscular atrophy in mouse

28 July 2018

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Direct central nervous system delivery provides enhanced protection following vector mediated gene replacement in a severe model of spinal muscular atrophy

28 July 2018

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28 July 2018

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Bifunctional RNAs targeting the intronic splicing silencer N1 increase SMN levels and reduce disease severity in an animal model of spinal muscular atrophy

28 July 2018

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28 July 2018

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28 July 2018

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Preclinical differences of intravascular AAV9 delivery to neurons and glia: a comparative study of adult mice and nonhuman primates

28 July 2018

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28 July 2018

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RETRACTED ARTICLE: Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN

28 July 2018

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28 July 2018

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28 July 2018

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A single nucleotide difference that alters splicing patterns distinguishes the SMA gene SMN1 from the copy gene SMN2.

28 July 2018

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28 July 2018

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28 July 2018

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Identification of proximal spinal muscular atrophy carriers and patients by analysis of SMNT and SMNC gene copy number

28 July 2018

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28 July 2018

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The genetic component in child mortality

28 July 2018

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28 July 2018

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28 July 2018

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27 September 2018

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27 September 2018

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27 September 2018

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Comparison of adeno-associated viral vector serotypes for spinal cord and motor neuron gene delivery

27 September 2018

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Intravenous scAAV9 delivery of a codon-optimized SMN1 sequence rescues SMA mice

27 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4351452

Systemic delivery of scAAV9 expressing SMN prolongs survival in a model of spinal muscular atrophy

27 September 2018

1 reference

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Regulation of SMN protein stability

27 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4351452

Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes

27 September 2018

1 reference

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Molecular analysis of spinal muscular atrophy and modification of the phenotype by SMN2.

27 September 2018

1 reference

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The Trendelenburg position increases the spread and accelerates the onset of epidural anesthesia for Cesarean section.

27 September 2018

1 reference

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Natural history in proximal spinal muscular atrophy. Clinical analysis of 445 patients and suggestions for a modification of existing classifications

27 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4351452

Reversible molecular pathology of skeletal muscle in spinal muscular atrophy

27 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4351452

SMN oligomerization defect correlates with spinal muscular atrophy severity

16 November 2018

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/25358252

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

SMNDelta7, the major product of the centromeric survival motor neuron (SMN2) gene, extends survival in mice with spinal muscular atrophy and associates with full-length SMN

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/25358252

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Newborn and carrier screening for spinal muscular atrophy

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/25358252

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.1038/MT.2014.210

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25358252%20AND%20SRC:MED&resulttype=core&format=json

7 June 2020

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25358252%20AND%20SRC:MED&resulttype=core&format=json

7 June 2020

25358252

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25358252%20AND%20SRC:MED&resulttype=core&format=json